NCT04428281

A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)

An Open-label, Multicenter Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of RO7248824 in Participants With Angelman Syndrome

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 74 (actual)
• Sponsor: Hoffmann-La Roche · Industry
• Sex: All
• Age: 1 Year – 12 Years
• Healthy volunteers: Not accepted

Summary

This is a phase I, multicenter, non-randomized, adaptive, open-label, multiple ascending, intra-participant, dose-escalation study with a long-term extension (LTE) part and an optional open-label extension (OOE) part. The objective of the study is to investigate the safety, tolerability, PK and PD of RO7248824 administered intrathecally (IT) in participants with AS. Two linked sets of dose escalation cohorts are planned based on two different age groups, namely participants with AS aged ≥ 5 to ≤ 12 years in cohorts A1 to A5 (with at least 2 participants ≤ 8 years old in each cohort) and AS participants aged ≥ 1 to ≤ 4 years in cohorts B1 to B5. The two sets of cohorts will be run in parallel, with each cohort A1 to A5 preceding and gating the linked cohort B1 to B5 (e.g., A1 precedes B1).

Conditions

• Angelman Syndrome

Interventions

Type	Name	Description
DRUG	RO7248824	In the MAD part, RO7248824 will be administered as IT injection of varing dose levels over a period of 8 weeks, with a minimum of approximately 4 weeks between each dose administration. In the LTE part, RO7248824 will be administered as IT injection of varying dose levels over a period of 144 weeks, with a minimum of approximately 16 weeks between each dose administration. In the OOE part, participants will receive RO7248824 as an IT injection with the same dosing regime as the LTE part over a period of up to 48 weeks.

Timeline

• Start date: 2020-08-19
• Primary completion: 2025-07-31
• Completion: 2025-07-31
• First posted: 2020-06-11
• Last updated: 2025-08-24

Locations

12 sites across 4 countries: United States, Italy, Netherlands, Spain

Regulatory

• FDA-regulated drug study